Presentation (CTI)

OBJECTIVES: Individuals with rare diseases often face inequalities in their care compared to the general population, including delayed diagnosis, uncoordinated care and limited access to effective treatment. National Institute for Health and Care Excellence (NICE) Highly Specialised Technology (HST) evaluations employ specific considerations and flexibilities to assess treatments for ultra-rare diseases. This research aims to identify the equality and equity factors that stakeholders consider important for HSTs, and those influencing NICE decision-making.
METHODS: All HST appraisals published since 2022 were included. Information was extracted from four stages of the HST process: company and stakeholder submissions, equality impact assessments, public committee slides and final guidance.
RESULTS: Nineteen HSTs were identified; all referenced equality/equity considerations, totalling 61 different considerations. Most issues related to the eligible population (36%, 22/61) and intervention uptake (33%, 20/61); 59% (36/61) were related specifically to the intervention. Some factors (30%, 18/61) were not addressed by or directly relevant to the treatment, such as issues regarding diagnosis. Most equity/equality considerations were discussed in the company submission (38%, 23/61) or committee slides (36%, 22/61) rather than the equality impact assessment (16%, 10/61). A total of 15 equity/equality issues were considered in the final guidance; 20% (3/15) were issues addressed by the intervention, 47% (7/15) were issues arising from the introduction of the intervention, and the remaining were broader considerations not directly related to the intervention (e.g. regarding the condition). Reporting did not indicate to what extent considerations influenced NICE’s decision-making. NICE highlighted that recommendations cannot be made outside of an intervention’s licence, limiting how equality/equity considerations, such as those regarding the eligible population, can be addressed.
CONCLUSIONS: While equality and equity considerations were frequently raised at various stages of HST evaluations, most were not reflected in NICE’s final guidance. Many considerations were deemed beyond the scope of the intervention or limited by its licence.